# US 10,869,853 B2 · Compositions and methods for treating autism spectrum disorders

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- Number: US 10,869,853 B2
- Document ID: US10869853B2
- Status: Granted
- Kind: B2
- Application number: 15/524,334
- Filed: 2015-10-02
- Published: 2017-11-30
- Granted: 2020-12-22
- Earliest claimed: 2011-10-31
- Applicants: The Johns Hopkins University; Antony Talalay
- Inventors: Paul Talalay; Andrew Zimmerman
- Web page: https://patentgrep.com/patent/US10869853B2

## CPC

- A61K 31/26: Cyanate or isocyanate esters; Thiocyanate or isothiocyanate esters
- A23L 33/105: Plant extracts, their artificial duplicates or their derivatives
- A23L 33/40: {Complete food formulations for specific consumer groups or specific purposes, e.g. infant formula}
- A61K 31/20: having a carboxyl group bound to a chain of seven or more carbon atoms, e.g. stearic, palmitic, arachidic acids
- A61K 31/53: having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine (melarsoprol A61K31/555 {; with four nitrogen atoms A61K31/495})
- A61K 31/55: having seven-membered rings, e.g. azelastine, pentylenetetrazole
- A61K 31/70: Carbohydrates; Sugars; Derivatives thereof (sorbitol A61K31/047)
- A61K 31/7048: having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin {, digitoxin or digoxin}
- A61K 36/31: Brassicaceae or Cruciferae (Mustard family), e.g. broccoli, cabbage or kohlrabi
- A61P 25/08: Antiepileptics; Anticonvulsants
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61K 2236/30: Extraction of the material
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A23L: FOODS, FOODSTUFFS OR NON-ALCOHOLIC BEVERAGES, NOT OTHERWISE PROVIDED FOR; PREPARATION OR TREATMENT THEREOF (preservation thereof A23B)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

The instant disclosure features, among other things, compositions and methods for treating an autism spectrum disorder in a human. The compositions comprise an effective amount of: (1) an isothiocyanate (e.g., sulforaphane or a derivative thereof) or (2) a glucosinolate, and optionally, an enzyme, to thereby treat an autism spectrum disorder and/or reduce the severity of at least one symptom of the disorder. Methods for preparing such compositions are also featured.

## Claims

### Claim 1

A method for treating an autism spectrum disorder in a human, the method comprising administering to the human daily a composition comprising an effective amount of an isothiocyanate, to thereby treat the disorder, wherein the effective amount is: between 25 and 75 μmol if the human weighs 100 pounds or less; between 75 and 125 μmol if the human weighs between 101 to 199 pounds; or between 125 and 175 μmol if the human weighs more than 200 pounds.

### Claim 2 (depends on 1)

The method of claim 1, wherein the amount is 50 μmol, if the human weighs 100 pounds or less; 100 μmol if the human weighs between 101-199 pounds; or 150 μmol if the human weighs more than 200 pounds.

### Claim 3 (depends on 1)

The method of claim 1, wherein the amount is administered daily as one dose.

### Claim 4 (depends on 1)

The method of claim 1, wherein the amount is administered daily as more than one dose.

### Claim 5 (depends on 1)

The method of claim 1, wherein the isothiocyanate is administered to the human for at least 18 weeks.

### Claim 6 (depends on 1)

The method of claim 1, wherein the disorder is autism.

### Claim 7 (depends on 6)

The method of claim 6, wherein the human has moderate to severe autism.

### Claim 8 (depends on 1)

The method of claim 1, wherein the amount is effective to reduce the severity of one or more behavioral symptoms of the disorder.

### Claim 9 (depends on 1)

The method of claim 1, wherein the human has a medical history of behavioral improvements with fever.

### Claim 10 (depends on 1)

The method of claim 1, wherein the isothiocyanate is sulforaphane or a derivative thereof.

### Claim 11 (depends on 10)

The method of claim 10, wherein the sulforaphane is enantiopure for the (R) enantiomer.

### Claim 12 (depends on 1)

The method of claim 1, wherein the isothiocyanate has been extracted from a cruciferous vegetable or seeds thereof.

### Claim 13 (depends on 1)

The method of claim 1, wherein the isothiocyanate has any one of the structural formulas (II) to (LII).

### Claim 14 (depends on 1)

The method of claim 1, wherein the isothiocyanate has at least 50% of the activity of an equivalent molar amount of sulforaphane to induce expression of a heat shock protein in cells in vitro.

### Claim 15 (depends on 1)

The method of claim 1, wherein the composition is a pharmaceutical composition.

### Claim 16 (depends on 1)

The method of any claim 1, wherein the composition is a medical food or a dietary supplement.

### Claim 17 (depends on 1)

The method of claim 1, wherein the composition is a food.

### Claim 18

A method for treating an autism spectrum disorder in a human, the method comprising administering to the human an effective amount of an isothiocyanate to thereby treat the disorder, wherein the human has not experienced a seizure within one year prior to administering the isothiocyanate.

### Claim 19

A method for treating an autism spectrum disorder in a human, the method comprising administering to the human: (a) an effective amount of an isothiocyanate to thereby treat the disorder; and (b) an effective amount of an anti-seizure drug.

### Claim 20

A method for treating an autism spectrum disorder in a human, the method comprising administering to the human an effective amount of an isothiocyanate to thereby treat the disorder, wherein the human is not concurrently being treated with a drug associated with increased risk of seizure.

### Claim 21

A method for treating an autism spectrum disorder in a human, the method comprising:
(i) administering to the human an effective amount of an isothiocyanate to thereby treat the disorder; and
(ii) monitoring the human for the occurrence of seizures.

### Claim 22

A method for treating an autism spectrum disorder in a human, the method comprising administering to the human an effective amount of an isothiocyanate to thereby treat the disorder, wherein the isothiocyanate is to be administered in conjunction with a reduced calorie diet.
